What is AMVUTTRA (vutrisiran)?
AMVUTTRA (vutrisiran) is a prescription medication developed by Alnylam Pharmaceuticals. It is indicated for the treatment of the polyneuropathy (nerve damage) associated with hereditary transthyretin-mediated amyloidosis (hATTR) in adults [1.6.9]. In early 2025, it also gained FDA approval to treat the cardiomyopathy (heart muscle disease) of both wild-type and hereditary transthyretin-mediated amyloidosis (ATTR-CM) [1.6.6].
This condition is a rare, progressive, and fatal disease caused by the misfolding of the transthyretin (TTR) protein [1.6.6]. These misfolded proteins accumulate as amyloid deposits in various organs, primarily the nerves and heart, leading to debilitating symptoms and organ failure [1.6.1, 1.6.6].
How AMVUTTRA Works: A Gene-Silencing Approach
AMVUTTRA is a pioneering RNA interference (RNAi) therapeutic, sometimes called a "gene silencer" [1.6.5]. It works by targeting and breaking down the messenger RNA (mRNA) responsible for producing both the normal (wild-type) and mutated TTR protein in the liver [1.6.3, 1.6.5]. By reducing the production of the TTR protein, AMVUTTRA decreases the buildup of harmful amyloid deposits, addressing the underlying cause of the disease [1.6.5, 1.6.6]. This mechanism helps prevent the progression of nerve and heart damage and can improve function and quality of life [1.6.5].
How much does AMVUTTRA cost per injection?
The list price of AMVUTTRA is substantial. The annual wholesale acquisition cost (WAC) is reported to be between $463,500 and $477,404 [1.3.6, 1.3.5]. AMVUTTRA is administered as a subcutaneous injection once every three months (quarterly) by a healthcare professional [1.4.6, 1.6.3].
Based on the annual price of approximately $476,000 and a quarterly dosing schedule, the cost per injection of AMVUTTRA is about $119,000 [1.2.1]. This high price point has been a subject of discussion, especially as it is significantly higher than some competing oral therapies [1.3.3, 1.3.4]. The manufacturer justifies the cost by highlighting its novel mechanism of action, convenient quarterly dosing, and clinical benefits [1.3.1, 1.3.5].
Important Safety Information and Side Effects
Before starting treatment, patients should be aware of key safety information. AMVUTTRA treatment leads to a decrease in serum vitamin A levels. Therefore, patients are advised to take the recommended daily allowance of a vitamin A supplement [1.6.4, 1.6.8].
The most common adverse reactions reported in clinical trials for hATTR-PN included:
- Pain in an extremity (15%) [1.6.4, 1.6.7]
- Arthralgia (joint pain) (11%) [1.6.4, 1.6.7]
- Dyspnea (shortness of breath) (7%) [1.6.4, 1.6.7]
- Decreased Vitamin A levels (7%) [1.6.4, 1.6.7]
Patients may also experience mild and temporary injection site reactions [1.6.3].
Comparison of ATTR Amyloidosis Treatments
AMVUTTRA is not the only treatment available for ATTR amyloidosis. It competes with other medications that have different mechanisms, administration methods, and costs.
| Feature | AMVUTTRA (vutrisiran) | ONPATTRO (patisiran) | TEGSEDI (inotersen) | VYNDAQEL/VYNDAMAX (tafamidis) |
|---|---|---|---|---|
| Mechanism | RNAi (Gene Silencer) [1.3.4] | RNAi (Gene Silencer) [1.4.8] | Antisense Oligonucleotide | TTR Stabilizer [1.3.7] |
| Administration | Subcutaneous Injection [1.4.5] | IV Infusion [1.4.4] | Subcutaneous Injection [1.4.3] | Oral Capsule (Pill) [1.3.3] |
| Frequency | Once every 3 months [1.6.3] | Once every 3 weeks [1.4.4] | Once weekly [1.4.3] | Once daily [1.3.1] |
| Annual Cost | ~$476,000 [1.3.1] | ~$463,500 [1.4.4] | ~$431,000 (approx. based on monthly) [1.4.1] | ~$244,000 - $250,000 [1.3.1, 1.3.2] |
Navigating the Cost: Alnylam Assist Program
Despite the high list price, many patients do not pay the full amount out-of-pocket. Alnylam, the manufacturer, offers a comprehensive patient support program called Alnylam Assist® [1.5.1]. This program is designed to help patients access treatment by providing financial support and navigating insurance coverage [1.5.4].
Key features of Alnylam Assist® include:
- Commercial Copay Program: For eligible patients with commercial insurance, this program can cover out-of-pocket costs. Alnylam states that the majority of commercially insured patients pay $0 for the medication [1.2.4, 1.5.5].
- Patient Assistance Program (PAP): This program may provide AMVUTTRA at no cost to eligible patients who are uninsured or underinsured [1.5.2, 1.5.5].
- Coverage Support: Case managers help patients understand their insurance benefits, handle prior authorizations, and investigate coverage options [1.5.1, 1.5.6].
- Quick Start Program: This program may provide the first dose at no cost for eligible patients experiencing delays in insurance approval [1.5.3].
It is important to note that patients with government-sponsored insurance like Medicare or Medicaid are not eligible for the commercial copay program [1.5.1]. However, AMVUTTRA is covered under Medicare Part B, and case managers can help patients explore other potential financial assistance options [1.5.8].
Conclusion
AMVUTTRA represents a significant advancement in treating ATTR amyloidosis, offering a convenient quarterly dosing schedule and a unique gene-silencing mechanism. While the answer to 'How much does AMVUTTRA cost per injection?' reveals a figure around $119,000, this list price is often not what patients pay. Through robust patient assistance programs like Alnylam Assist®, the manufacturer aims to make the treatment accessible and affordable for most eligible individuals, mitigating the barrier of its high initial cost [1.5.7]. Patients considering AMVUTTRA should speak with their healthcare provider and contact Alnylam Assist® to fully understand their potential costs and available support [1.5.6].
For more information on financial support, visit the official Alnylam Assist® website [1.5.1].
